In The Know: Analyst answers — The gene therapy revolution

As gene therapies for major diseases accelerate from the development pipeline, how can pharma exploit near-term opportunities and protect its business going forward? Investigating the challenges pharma will face along the way, from choosing the right target, mastering commercialisation, and getting the product out to the patients effectively, we spoke to subject expert and analyst Dr Cheryl Barton to dive into recent research. Here she shares her thoughts on the future evolution of gene therapies.

FirstWord: What makes the topic of gene therapy an important area for pharmaceutical companies right now?

Cheryl Barton: Having conducted several rounds of research on the topic in the last 18 months, there is no doubt that this is a maturing market, as it has experienced significant changes in a short time frame. Today, gene therapy continues to evolve as we are seeing more clinically proven outcomes. Pharma is now shifting the focus to fine tuning their delivery methods, generating durable responses and continuing to introduce new products into the market. We see this as the European market has opened up, approving CAR-T treatments and accessing gene therapies for orphan indications. In the last year, there has been quite a lot of activity around gene therapy and pharma companies are coming to realise that these treatments can be complementary to their portfolio of products in development.

FW: What experts have you spoken with on the subject?

CB: Each of the four experts I interviewed have a good pulse on the investment and commercial strategies needed to better understand today’s gene therapy market:

● Raymond T. Bartus, President at RTBioconsultants. Bartus received a PhD in the neurosciences at North Carolina State University and has since focused his career on translational R&D in industry, while also maintaining strong academic and scientific ties.

● Miguel Forte is the Chief Executive Officer of Zelluna Immunotherapy and currently Chief Commercialisation Officer and Chair of the Commercialisation Committee, International Society of Cellular Therapy. Forte has extensive expertise in the regenerative medicine and cell therapy industries, as well as medical and regulatory affairs.

● Dr Joachim Fruebis is Senior Vice President of Development at Bioverativ. Fruebis is an experienced leader with a long history of developing novel therapies in multiple disease areas in the pharmaceutical and biotechnology industry. He is currently responsible for the build up and development of the Bioverativ programme portfolio.

● Sander van Deventer is the Chief Scientific Officer of uniQure and has had a critical role in the development of the first commercial monoclonal antibody (Remicade; infliximab) and the first gene therapy to be granted market authorisation in the Western world (Glybera).

FW: Which key issue generated the most interest during conversations with experts?

CB: In speaking with the experts, there are multiple issues to overcome, such as product manufacturing and distribution, and reducing potential immunogenicity. Additionally, it has been recently noted by the FDA commissioner that these evolving products require about 20 percent dedicated time for development and 80 percent for manufacturing. This claim highlights the fact that there is still room for improvements to make gene therapies more cost efficient in the years to come.

FW: What was one of the most insightful interview quotes? What did it teach us?

CB: One expert compared gene therapy to the monoclonal antibody market, which has had numerous failures, but eventually was successful in becoming the multibillion dollar market it is today:

“The point is some diseases are far too complicated to necessarily expect expression of a single gene product to effectively treat the disease, particularly in the initial clinical efforts. If you look back at antibody therapy 40 years ago, there was a lot of hype, many companies came and went – with far more failures than successes. Eventually, that field matured as new and important information was gradually assimilated, allowing antibodies to become a multibillion-dollar franchise for the biotech/pharmaceutical industry. But it took a long time to get to that point.” [US Industry Expert]

FW: What was the most notable takeaway from researching the impact of genomics?

CB: Gene therapies are not without their challenges. According to the experts, there are still significant advancements to be made in their delivery and manufacturing processes. However, the key driver to push the industry forward will be focusing on disease areas with unmet needs that have not been answered by traditional routes. Therefore, the next wave of therapies will be targeting non-orphan conditions such as haemophilia, as well as addressing the unmet needs of certain cancers and neurological diseases.